Agreement formed for development of AAV vectors for ocular gene therapy
German-based company, SIRION Biotech has signed a two-year development agreement with a clinical-stage ophthalmology company, Acucela, to establish optimised adeno associated virus (AAV) vectors for clinical applications in ocular gene therapy.
Under the terms of the agreement, both companies will collaborate to find a genetic cure for retinitis pigmentosa (RP) and SIRION will receive milestone payments with additional payments to be made on net sales from resulting products or therapies.
RP is an inherited problem causing retinal degeneration, leading to complete blindness. Currently, there are no pharmacological treatments available for RP, with recombinant AAV vectors considered to be promising as a gene delivery system for therapeutic applications — pre-clinical experiments have demonstrated successful delivery of human rhodopsin (hRho) to the retina.
Through this partnership, and in conjunction with an academic partner consortium, the companies hope to develop the next generation of recombinant AAV vectors. The goal will be to secure new and modified AAV capsids to ensure that the therapeutic viral particles exhibit a safe product profile with improved specificity for therapeutic protein delivery, over wild type vectors, needed to effectively restore light sensitivity to patients.
“The academic acumen of our partners, together with our strong viral vector specialisation and experience will empower our client to enter clinical trials with an efficient, safe and scalable product. For us it is the chance to participate in a fundamental step for gene therapies that can help millions of patients worldwide win back their eyesight. This is what we started SIRION Biotech for,” said Dr Christian Thirion, founder and CEO of the company.
Dr Ryo Kubota, PhD, chairman, president and CEO of Acucela added, “We are pleased to advance our optogenetic genetherapy programme in partnership with SIRION Biotech to bring a new therapeutic approach to RP patients suffering from this devastating disease.”
SIRION Biotech provides custom engineering services of viral vector strategies for research and development in the life sciences and industry.
Acucela is a wholly-owned subsidiary of Kubota Pharmaceutical Holdings and is focused on developing a diverse portfolio of drugs and devices to preserve and restore vision.